Next Topics

CAR-T Therapies


ICER will develop a report on chimeric antigen receptor t-cell (CAR-T) therapies for treatment of B-cell malignancies. ICER’s report will consider the comparative clinical effectiveness and value of tisagenlecleucel (KymriahTM , Novartis) and axicabtagene ciloleucel (YescartaTM, Kite Pharma). Both agents were approved in 2017. Tisagenlecleucel is approved for treatment of relapsed or refractory B-cell acute lymphoblastic leukemia in pediatric and young adult patients. Novartis is also expected to file later this year for a second indication for diffuse large B-cell lymphoma in adults. Axicabtagene ciloleucel is approved for the treatment of relapsed or refractory aggressive non-Hodgkin’s lymphoma in adult patients.

Date of Review: March 2018

Hemophilia A

New England CEPAC

The Institute for Clinical and Economic Review (ICER) will develop a report assessing the comparative clinical effectiveness and value of emicizumab (Hemlibra®, Genentech), a monoclonal antibody currently under review by the FDA for prevention of bleeding in patients who have hemophilia A with coagulation factor VIII inhibitors. Factor VIII is the clotting protein that is deficient in hemophilia A, and inhibitors interfere with factor replacement therapy. Emicizumab was approved by the FDA in November of 2017.

Cystic Fibrosis

Midwest CEPAC

Cystic fibrosis is an autosomal recessive condition caused by mutations in the CFTR gene,. It is relatively rare, occurring in approximately 1 in 2,500 to 3,000 livebirths, but is the most common, lethal genetic disease in Caucasian populations. CF is a progressive disease that affects many organ systems, but most of its morbidity and mortality are associated with its impact on the respiratory system.

Interventions of Interest:

  • Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals)
  • Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals)
  • Ivacaftor (Kalydeco®, Vertex Pharmaceuticals)



Migraine is a common, recurrent headache disorder that affects approximately 18% of women and 6% of men in the United States (US). Migraine is among the top ten causes of years lived with disability in the US. When a person experiences a migraine, she or he may feel moderate-to-severe pain and other symptoms (e.g., nausea, vomiting, or sensitivity to light or to sound), have a reduced ability to function, or require bed rest. Despite its high prevalence and impairment, migraine is often not recognized or effectively treated.

Interventions of interest, July 2018 review:

  • Erenumab (Amgen/Novartis)
  • Fremanezumab (Teva)
  • Galcanezumab (Eli Lilly)

Interventions of interest, July 2014 review:

  • OnabotulinumtoxinA (Botox®, Allergan, Inc)
  • Transcutaneous electrical nerve stimulation (Cefaly®, Cefaly Technology)
  • Single pulse transcranial magnetic stimulation (SpringTMS™, eNeura Inc.)



New England CEPAC

Plaque psoriasis is a common disease that causes red, scaly, raised lesions on the skin, most commonly on the elbows, knees, scalp, and back. Psoriasis affects about 2% of the population and significantly decreases health-related quality of life, particularly if lesions are in areas that can affect daily functioning (e.g., the hands or soles of the feet) or affect social functioning (e.g., the face). Psoriasis is a chronic inflammatory condition that is associated with systemic diseases including psoriatic arthritis, other autoimmune diseases, the metabolic syndrome, and cardiovascular disease.

Interventions of interest:

  • Anti TNF agents: Adalimumab (Humira®, AbbVie), etanercept (Enbrel®, Amgen, Inc.), infliximab (Remicade®, Janssen)
  • Anti IL-17A agents: Secukinumab (Cosentyx®, Novartis), ixekizumab (Taltz®, Eli Lilly and Co.), brodalumab (SiliqTM, Valeant Pharmaceuticals and AstraZeneca)
  • Anti IL-12/23 agent: Ustekinumab (Stelara®, Janssen)
  • Anti PDE4 agent: Apremilast (Otezla®, Celgene)

Included in planned 2018 Condition Update:

  • Anti IL-23 agents: Guselkumab (Tremfya®, Janssen/Johnson & Johnson), tildrakizumab (Sun Pharma)
  • TNF inhibitor: Certolizumab pegol (Cimzia®, UCB)


New England CEPAC

ICER will review elagolix (AbbVie) for management of endometriosis with associated pain.

Prostate Cancer


Ongoing Review

ICER will develop a report on anti-androgen drugs for high risk prostate cancer. The report is expected to include enzalutamide (Xtandi®, Astellas and Medivation), abiraterone acetate (Zytiga®, Janssen), apalutamide (Erleada™, Janssen).

Past Reviews

  • September 2009: Active surveillance and Radical prostatectomy 
  • December 2008: Brachytherapy and Proton beam therapy
    Proton beam therapy was also the subject of an evidence review by ICER for the Washington State Health Care Authority in May 2014.
  • November 2007: Intensity modulated radiation therapy (IMRT)


Midwest CEPAC

ICER will review new therapies for the treatment of hereditary transthyretin-related (hATTR) amyloidosis, a rare genetic condition characterized by nerve, heart, and eye damage that is currently treated through supportive measures alone.

The review is expected to include inotersen (Ionis Pharmaceuticals, Inc.), an antisense oligonucleotide that interferes with transthyretin production in the liver, and patisiran (Alnylam Pharmaceuticals), which inhibits transthyretin through an RNA interference pathway. Approvals are expected in July and August of 2018, respectively.

past Topics

Jan 2018
Midwest CEPAC

Voretigene Neparvovec

ICER will develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (LuxturnaTM, Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be approved by the US Food and Drug Administration (FDA).

Date of review: January 2018

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Dec 2017
New England CEPAC

Tardive Dyskinesia

Tardive dyskinesia is a serious and disabling movement disorder that affects individuals treated with antipsychotic agents for a variety of mental health conditions. Though prevention and treatment primarily focus on stopping or changing the offending antipsychotic agent, for some individuals there are no other effective treatment options, and symptoms can persist after discontinuing or changing antipsychotic therapy.

Interventions of Interest:

  • Valbenazine (Ingrezza®, Neurocrine Biosciences, Inc)
  • Deutetrabenazine (Austedo®, Teva)
  • Tetrabenazine (Xenazine®)

Date of Review: December 2017

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Oct 2017
CTAF, ICER, Washington State

Low Back Pain

ICER will develop a report assessing the comparative clinical effectiveness and value of certain non-pharmacologic, non-invasive interventions for chronic low back and neck pain. The report will be the subject of a October 19, 2017 meeting of the California Technology Assessment Forum.

Interventions of Interest:

  • Acupuncture
  • Cognitive behavioral therapy
  • Mindfulness
  • Yoga
  • Tai Chi

Date of review: October 2017

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Sep 2017
Midwest CEPAC

Ovarian Cancer

ICER’s report reviews evidence on the comparative clinical effectiveness and value of poly (ADP-ribose) polymerase (PARP) inhibitors for treatment of ovarian cancer.

Interventions of Interest:

  • Rucaparib (Rubraca®, Clovis Oncology)
  • Niraparib (Zejula®, Tesaro, Inc.)
  • Olaparib (Lynparza®, AstraZeneca)
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Jul 2017
New England CEPAC

Abuse-Deterrent Opioids

Opioids are used to treat cases of acute and chronic pain that arise from a variety of causes, ranging from trauma to palliative care for advanced illness. Every year, 100 million people in the United States suffer from pain, 9-12 million of whom have chronic or persistent pain. Although opioid therapy is an important component of pain management for many patients, the addictive and euphoric properties of these drugs make them liable to misuse, abuse, addiction, and diversion.

In an effort to help tackle the public health crisis of opioid dependence, misuse, and addiction that has emerged over the last decade, drug manufacturers have begun to develop abuse-deterrent formulations of opioid medications (ADFs). ICER will review available evidence on the comparative clinical effectiveness and comparative value of ADFs in order to inform decision-making by patients, clinicians and policy-makers, within the context of multiple efforts being undertaken to combat the opioid crisis. ICER’s review will also analyze the potential of ADFs to reduce the burden of prescription opioid abuse by evaluating their benefits and market penetration based on coverage policies and legislation.

Interventions of Interest:

  • Hysingla® ER (Hydrocodone, Purdue)
  • Vantrela® (Hydrocodone , Teva)
  • Arymo® ER ( Morphine, Egalet)
  • Embeda® (Morphine + naltrexone, Pfizer)
  • MorphabondTM (Morphine extended release, Inspirion Delivery Technologies)
  • OxyContin® TR (Oxycodone, Purdue)
  • Xtampza®ER (Oxycodone, Collegium Pharmaceutical Inc)
  • Targiniq® (Oxycodone + naloxone extended release, Purdue)
  • Troxyca® ER (Oxycodone + naltrexone, Pfizer)
  • RoxyBond® (Oxycodone, Inspirion Delivery Technologies)

Date of Review: July 2017

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Jun 2017


Osteoporosis, the weakening of the bones through loss of bone mineral content and a decrease in bone quality, is a common disease of aging that affects approximately 10 million Americans. Approximately half of women and one quarter of men will suffer at least one fracture due to osteoporosis during their lifetimes. Experts estimate that there are approximately two million osteoporotic fractures each year, which results in $19 billion in related costs. By 2025, these figures are predicted to grow to approximately three million fractures and $25 billion in costs annually as the population of older Americans increases.

Interventions of Interest:

  • Abaloparatide (Radius Health, Inc.)
  • Teriparatide (Forteo®, Eli Lilly and Co.)
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May 2017

Orphan Drug Summit

ICER hosted a multi-stakeholder policy summit to discuss methods for assessing the value of new drugs for rare conditions. The summit included discussion of how these methods can be applied to recommend fair prices that reflect the value of orphan drugs to patients and the health system to allow for broader insurance coverage for innovative new treatments.

More information about the summit is available in a press release.

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May 2017
Midwest CEPAC

Atopic Dermatitis

Atopic dermatitis (eczema) is a chronic/chronically-relapsing skin condition characterized by itching and dry skin. Atopic dermatitis is common. It affects 5-20% of children worldwide, and approximately 11% of children in the US. It is also estimated to affect around 3-7% of adults in the US. Management of atopic dermatitis can create burdens for the family, and the disorder can decrease quality of life. Itching, in particular, can disrupt sleep and lead to daytime sleepiness, irritability, and psychological stress, and cosmetically important lesions can lead to social stress and isolation.

ICER’s report will evaluate the comparative clinical effectiveness and value of two emerging therapies for atopic dermatitis: crisaborole and dupilumab. Crisaborole will be evaluated based on its expected indication in the treatment of mild-to-moderate atopic dermatitis in children and adults; separately, dupilumab will be evaluated for its expected indication in the treatment of moderate-to-severe atopic dermatitis in adults.

Interventions of Interest:

  • Crisaborole (Eucrisa™, Pfizer, Inc.)
  • Dupilumab (Dupixent®, Sanofi and Regeneron, Inc.)

Date of Review: May 2017

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Mar 2017
New England CEPAC


Rheumatoid arthritis (RA) is the most common autoimmune inflammatory arthritis in adults, affecting approximately 1.5 million Americans. RA is more common in women and may occur at any age, with peak incidence occurring at ages 50-60 years. RA is typically characterized by morning stiffness and symmetrical joint swelling of the feet, hands, and knees, although any joint (and in some cases, internal organs) may be involved. The course of RA may be complicated by cardiac, hematologic, and other extra-articular manifestations.

Interventions of Interest:

TNF Inhibitors

  • Adalimumab (Humira®, Abbvie)
  • Certolizumab pegol (Cimzia®, UCB)
  • Etanercept (Enbrel®, Amgen)
  • Golimumab (Simponi®, Janssen)
  • Infliximab (Remicade®, Janssen)

CD20- directed cytolytic antibody

  • Rituximab (Rituxan®, Genentech)

Tcell inhibtor

  • Abatacept (Orencia®, BMS)

IL-6 inhibitors

  • Sarilumab (Kevzara®, Sanofi and Regeneron Pharmaceuticals)
  • Tocilizumab (Actemra®, Genentech)

JAK- inhibitors

  • Tofacitinib (Xeljanz®, Pfizer)
  • Baricitinib (Eli Lilly and Co.)

Date of Review: March 2017


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Feb 2017

Multiple Sclerosis

Multiple sclerosis (MS) is a chronic, immune-mediated inflammatory demyelinating disease of the central nervous system (CNS). Approximately 400,000 Americans have MS, although this may be an underestimate, and the disease affects about twice as many women as men. Some patient groups, such as African Americans, experience a more rapid and severe clinical course. MS is the most common nontraumatic cause of permanent disability among young adults. The annual cost of MS in the United States is estimated to be $28 billion.

ICER’s review will incorporate treatments for two types of MS. The most common form of the disease is relapsing-remitting MS (RRMS), which affects 85% to 90% of patients with MS. RRMS is characterized by periodic relapses characterized by neurologic symptoms that diminish or resolve with treatment. The report will also review treatments for primary-progressive MS (PPMS), which affects approximately 10-15% of MS patients. PPMS is characterized by steadily worsening neurologic function without remissions or acute episodes.

Interventions of Interest:

Injectable Agents

  • Daclizumab (Zinbryta®, Biogen/AbbVie)
  • Glatiramer acetate (Copaxone®, Teva)
  • Glatiramer acetate (Glatopa®, Sandoz (Novartis))
  • Interferon beta-1a (Avonex®, Biogen)
  • Interferon beta-1b (Betaseron®, Bayer)
  • Interferon beta-1b (Extavia®, Novartis)
  • Interferon beta-1a (Rebif®, EMD Serono)
  • Peginterferon beta-1a (Plegridy®, Biogen)

Oral Agents

  • Dimethyl fumarate (Tecfidera®, Biogen)
  • Fingolimod (Gilenya®, Novartis)
  • Teriflunomide (Aubagio®, Sanofi-Genzyme)

Infused Agents

  • Alemtuzumab (Lemtrada®, Sanofi-Genzyme)
  • Natalizumab (Tysabri®, Biogen)
  • Ocrelizumab (Ocrevus®, Roche/Genentech)
  • Rituximab (Rituxan®, Roche/Genentech)

Date of Review: February 2017

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